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Rexgenero Announces Positive Outcome of Independent Data Monitoring Committee Meeting for Phase III Trial for Diabetic Patients with Critical Limb-Threatening Ischaemia

LONDON–()–Rexgenero, a regenerative medicine company developing advanced cell therapies to treat critical limb-threatening ischaemia (CLI), today announces that the Independent Data Monitoring Committee (IDMC) for its REX-001 phase III clinical trial recently met to review patient safety and tolerability data from the initial 22 subjects. Based on this review, the IDMC unanimously agreed to continue the trial unchanged due to no significant safety concerns.

“We are pleased to receive the IDMCs recommendation that our REX-001 phase III clinical study should continue without any protocol modifications. This achievement is an important milestone reflecting the safety profile of REX-001 in treating diabetic patients with critical limb-threatening ischaemia, a severe condition with high unmet need,” Gilbert Wagener, Senior Vice President, Chief Medical Officer at Rexgenero, said: “We thank our team and study investigators for their efforts in running the study, and the IDMC members for their supervision and thoughtful review of the patient data. Despite the unprecedented challenges of the COVID-19 pandemic, we believe we are back on track in terms of patient recruitment and moving the REX-001 phase III trial forward.”

CLI is the most serious form of chronic peripheral artery disease, in which severe obstruction of the arteries, as a result of plaque formation, markedly reduce blood flow to the extremities, mainly to the legs and feet. CLI may occur in one or both legs and it is characterized by excruciating pain at-rest, often including ulcers which may become gangrenous requiring amputation of the limb. The condition represents an area of high unmet medical need with very limited treatment options currently on the market, highlighting the importance of Rexgenero’s pipeline.

REX-001 consists of bone marrow-derived white blood cells extracted from the patient (autologous) and has shown efficacy in 80% of patients in early phase clinical studies. The phase III study is a pivotal, placebo-controlled, double-blind, parallel-group, adaptive trial conducted in subjects with diabetes and Rutherford Category 5 CLI. Patients are randomized in a 2:1 ratio to receive a single intra-arterial administration of REX-001 or matching placebo into the index limb.

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About Rexgenero

Rexgenero is a privately held, UK-based cell and gene therapy company pioneering the development of therapies to treat serious diseases such as chronic limb-threatening ischaemia (CLI), cancer and immunological disorders.

Rexgenero’s lead product, REX-001, consists of a suspension of immune and progenitor cells involved in immune modulation, blood vessel regeneration and remodelling and improvement in blood flow. It is manufactured using a patient’s own cells, avoiding undesired immune responses. REX-001 is currently being tested in pivotal, placebo-controlled, double-blind, adaptive Phase III SALAMANDER trials in patients with CLI and diabetes mellitus at multiple sites across Europe. Previous randomised REX-001 clinical trials have already demonstrated statistically significant proof of concept. In a Phase I/II and Phase II clinical trial, REX-001 showed very strong positive results in improvement of blood flow, healing of ulcers and alleviating chronic ischemic rest pain. All ulcers healed within 12 months in 82% of patients after treatment with REX-001 at the dose being currently tested in Phase III trials.

The company also has a pioneering synthetic gene delivery platform, including an off-the-shelf CAR-T therapy allowing the in vivo targeting and transduction of T-cells.

Rexgenero’s lentivector, encoding for the Chimeric Antigen Receptor, is coated with a polymeric envelope grafted with an anti-CD3 targeting moiety to target T-cells. In contrast to approved ex vivo CAR T-cell therapies, the company’s in vivo technology requires low-cost manufacturing. The genetic modification to express the CAR is generated directly in the patient. Rexgenero’s inducible, switchable, in vivo CAR-T platform, represents a significant advantage over current recently approved ex vivo CAR-T therapies, with the potential for more effective, more universal, and safer treatments.

Rexgenero has offices in London, UK with R&D and manufacturing operations in Seville, Spain and Paris, France and additional manufacturing capability in Frankfurt, Germany.

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About REX-001 and the Phase III SALAMANDER clinical trials

REX-001 consists of bone-marrow-derived white blood cells extracted from the patient (autologous) and has shown efficacy in 80% of patients in Phase II and I/II studies.

Rexgenero is in the process of treating a total of 138 patients with CLI and diabetes in two pivotal, placebo-controlled, double-blind and adaptive Phase III clinical trials (dubbed the ‘SALAMANDER’ clinical trials) at approximately 30 sites across Europe.

The clinical trials use a highly innovative and adaptive trial design with novel primary endpoints that increase the sensitivity of the study and increase the likelihood of REX-001 succeeding as a breakthrough treatment for CLI.

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